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Published on Aug 10, 2012
Since treatments for genetic diseases have been found there has been a major shift in acceptance of patient registries. APBD patients can be assured the registry would be maintained in an anonymous and protected way, ensuring confidentiality. Registries provide a natural history of a disease. If you can show you can alter the natural history of a genetic disorder favorably and improve quality of life by comparing it with the untreated state from a registry, then you're much better positioned to get FDA approval for new treatments and new drugs.